Costa Enrico, Magrini Nicola
Settore Affari Internazionali, Agenzia Italiana del Farmaco, Roma; Committee for Orphan Medicinal Products (COMP), Agenzia Europea dei Medicinali (EMA), Amsterdam.
Direttore Generale, Agenzia Italiana del Farmaco, Roma.
Recenti Prog Med. 2022 Jul-Aug;113(7):407-410. doi: 10.1701/3850.38333.
In the past few decades, the regulatory system has changed its approach to speed up the assessment and approval of drugs for the treatment of serious and orphan diseases. However, too early assessments may fail to provide solid evidence to define the therapeutic value of drugs and their fair price. As for rare diseases, patients' expectations amplify such criticalities due to the lack of therapeutic alternatives. The revision of the EU regulation on orphan medicinal products represents an opportunity to promote greater integration between the development and approval of drugs with their subsequent access. For this reason, we propose: 1) establishing a process for evaluating the comparative efficacy and quantification of the therapeutic benefit of a drug at a European level, while addressing incentives towards rarer diseases to support their development and marketing; 2) downsizing the impact of orphan drugs on general sustainability, considering that incentives supporting their development and marketing cannot transform them into new 'blockbusters'. In this perspective, a European procurement - initially intended for drugs for ultra-rare diseases - could improve access by all EU countries through simplifying the burdensome procedures for the pharmaceutical industry; 3) enhancing the contribution of post-authorization research from a public-private partnership perspective to steer the development of drugs already approved towards rarer pathologies or mutations of little commercial interest.
在过去几十年里，监管体系已改变其方式，以加快治疗严重疾病和罕见病药物的评估与审批。然而，过早的评估可能无法提供确凿证据来界定药物的治疗价值及其合理价格。对于罕见病而言，由于缺乏治疗选择，患者的期望加剧了此类关键性问题。欧盟孤儿药法规的修订是一个促进药物研发、审批及其后续可及性之间实现更大程度整合的契机。因此，我们提议：1）在欧洲层面建立一个评估药物相对疗效和量化治疗效益的流程，同时针对更罕见疾病设立激励措施，以支持其研发和上市；2）鉴于支持孤儿药研发和上市的激励措施无法将它们转变为新的“重磅炸弹”药物，应降低孤儿药对总体可持续性的影响。从这一角度看，一项最初针对超罕见病药物的欧洲采购计划，可通过简化制药行业繁琐的程序，改善所有欧盟国家的可及性；3）从公私合作的角度加强上市后研究的贡献，引导已获批药物朝着罕见病理或商业价值不大的突变方向发展。
